Ontology highlight
ABSTRACT:
SUBMITTER: Morgan RA
PROVIDER: S-EPMC6953778 | biostudies-literature | 2020 Jan
REPOSITORIES: biostudies-literature
Molecular therapy : the journal of the American Society of Gene Therapy 20190928 1
β-globin lentiviral vectors (β-LV) have faced challenges in clinical translation for gene therapy of sickle cell disease (SCD) due to low titer and sub-optimal gene transfer to hematopoietic stem and progenitor cells (HSPCs). To overcome the challenge of preserving efficacious expression while increasing vector performance, we used published genomic and epigenomic data available through ENCODE to redefine enhancer element boundaries of the β-globin locus control region (LCR) to construct novel E ...[more]